AAV vectors: The Rubik's cube of human gene therapy
A Pupo, A Fernández, SH Low, A François… - Molecular Therapy, 2022 - cell.com
Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …
Progranulin as a therapeutic target in neurodegenerative diseases
Progranulin (PGRN, encoded by the GRN gene) plays a key role in the development,
survival, function, and maintenance of neurons and microglia in the mammalian brain. It …
survival, function, and maintenance of neurons and microglia in the mammalian brain. It …
FSH blockade improves cognition in mice with Alzheimer's disease
Alzheimer's disease has a higher incidence in older women, with a spike in cognitive
decline that tracks with visceral adiposity, dysregulated energy homeostasis and bone loss …
decline that tracks with visceral adiposity, dysregulated energy homeostasis and bone loss …
Base editing rescue of spinal muscular atrophy in cells and in mice
Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, arises from
survival motor neuron (SMN) protein insufficiency resulting from SMN1 loss. Approved …
survival motor neuron (SMN) protein insufficiency resulting from SMN1 loss. Approved …
Glucose metabolism links astroglial mitochondria to cannabinoid effects
D Jimenez-Blasco, A Busquets-Garcia… - Nature, 2020 - nature.com
Astrocytes take up glucose from the bloodstream to provide energy to the brain, thereby
allowing neuronal activity and behavioural responses,,,–. By contrast, astrocytes are under …
allowing neuronal activity and behavioural responses,,,–. By contrast, astrocytes are under …
[HTML][HTML] Adeno-associated virus technologies and methods for targeted neuronal manipulation
Cell-type-specific expression of molecular tools and sensors is critical to construct circuit
diagrams and to investigate the activity and function of neurons within the nervous system …
diagrams and to investigate the activity and function of neurons within the nervous system …
Glucose-dependent insulinotropic polypeptide receptor-expressing cells in the hypothalamus regulate food intake
AE Adriaenssens, EK Biggs, T Darwish, J Tadross… - Cell metabolism, 2019 - cell.com
Ambiguity regarding the role of glucose-dependent insulinotropic polypeptide (GIP) in
obesity arises from conflicting reports asserting that both GIP receptor (GIPR) agonism and …
obesity arises from conflicting reports asserting that both GIP receptor (GIPR) agonism and …
CNS transduction benefits of AAV-PHP. eB over AAV9 are dependent on administration route and mouse strain
SN Mathiesen, JL Lock, L Schoderboeck… - … Therapy-Methods & …, 2020 - cell.com
Adeno-associated viral (AAV) vectors are attractive tools for central nervous system (CNS)
gene therapy because some vectors can cross the blood-brain barrier (BBB), allowing them …
gene therapy because some vectors can cross the blood-brain barrier (BBB), allowing them …
dCas9-based Scn1a gene activation restores inhibitory interneuron excitability and attenuates seizures in Dravet syndrome mice
Dravet syndrome (DS) is a severe epileptic encephalopathy caused mainly by heterozygous
loss-of-function mutations of the SCN1A gene, indicating haploinsufficiency as the …
loss-of-function mutations of the SCN1A gene, indicating haploinsufficiency as the …
Efficient strategies for microglia replacement in the central nervous system
Microglia are important immune cells in the central nervous system (CNS). Dysfunctions of
gene-deficient microglia contribute to the development and progression of multiple CNS …
gene-deficient microglia contribute to the development and progression of multiple CNS …